The Bluebird Bio product Zynteglo, a gene therapy used to treat beta-thalassemia in patients, received FDA approval on Wednesday.
Why it’s important The one-time procedure, which aids patients whose life expectancy is shortened by recurrent blood transfusions, costs a record-breaking $2.8 million.
It is the third FDA-approved gene therapy to date.
Approximately 1,300 to 1,500 people are thought to have beta-thalassemia, also referred to as beti-cel, according to Bluebird.
The business intends to employ an outcome-based agreement and has promised to refund insurers for up to 80% of the therapy’s cost for those patients who require routine transfusions even after receiving treatment.
What they say is this: Despite having a price tag of more than $2 million, the medicine has a high long-term value, according to a statement from the Institute for Clinical and Economic Review, an organization that assesses drug pricing and effectiveness.
The bottom line is that gene treatments hold great potential for the treatment of uncommon disorders. However, they have so far proven to be expensive, and the expanding list of these treatments could result in astronomical health care costs.